公开(公告)号：US12122995B2

公开(公告)日：2024-10-22

申请号：US16726163

申请日：2019-12-23

申请人： DNA2.0, INC.

发明人： Jeremy Minshull ,  Mark Welch ,  Sridhar Govindrajan ,  Maggie Lee ,  Kate Caves ,  Jon Ness

IPC分类号： C12N9/12 ,  C12N1/16 ,  C12N1/18 ,  C12N15/10 ,  C12N15/52 ,  C12N15/62 ,  C12N15/63 ,  C12N15/67 ,  C12N15/81 ,  C12N15/85 ,  C12N15/90 ,  C12R1/84 ,  C12R1/865

CPC分类号： C12N1/165 ,  C12N1/185 ,  C12N9/12 ,  C12N9/1241 ,  C12N15/1082 ,  C12N15/52 ,  C12N15/625 ,  C12N15/63 ,  C12N15/81 ,  C12N15/815 ,  C12N15/85 ,  C12N15/8509 ,  C12N15/90 ,  C12Y207/00 ,  C12Y207/07 ,  C07K2319/09 ,  C12N15/67 ,  C12N2800/90 ,  C12N2830/007 ,  C12N2830/40 ,  C12N2830/42 ,  C12N2840/203 ,  C12R2001/84 ,  C12R2001/865

摘要： The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

公开(公告)号：US10041077B2

公开(公告)日：2018-08-07

申请号：US15287624

申请日：2016-10-06

申请人： DNA2.0, INC.

发明人： Jeremy Minshull ,  Maggie Lee

IPC分类号： C12N15/00 ,  C07H21/02 ,  C12N15/67 ,  C12N15/52 ,  C12N15/81 ,  C12N15/85 ,  C12N9/12 ,  C12N15/62 ,  C12R1/84 ,  C12R1/865 ,  C12N15/10 ,  C12N15/63

CPC分类号： C12N15/67 ,  C12N9/12 ,  C12N9/1241 ,  C12N15/1082 ,  C12N15/52 ,  C12N15/625 ,  C12N15/63 ,  C12N15/81 ,  C12N15/815 ,  C12N15/85 ,  C12N15/90 ,  C12N2830/007 ,  C12N2830/40 ,  C12N2830/42 ,  C12N2840/203 ,  C12R1/84 ,  C12R1/865 ,  C12Y207/00

摘要： The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

公开(公告)号：US09827295B2

公开(公告)日：2017-11-28

申请号：US14889750

申请日：2014-05-15

申请人： Regents of the University of Minnesota ,  REGENXBIO Inc.

发明人： R. Scott McIvor ,  Lalitha R. Belur ,  Walter Low ,  Carolyn Fairbanks ,  Karen Kozarsky

IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N15/11 ,  C12N15/113 ,  C12N15/00 ,  C12N15/09 ,  C12N15/10 ,  A61K38/47 ,  C12N7/00

CPC分类号： A61K38/47 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/007 ,  C12Y302/01076

摘要： A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.

公开(公告)号：US09636354B2

公开(公告)日：2017-05-02

申请号：US14686541

申请日：2015-04-14

申请人： University of Florida Research Foundation, Inc.

发明人： David C. Bloom ,  Antonio L. Amelio

IPC分类号： C12N5/00 ,  A61K31/713 ,  C12N15/86 ,  C12N15/00 ,  A61K48/00

CPC分类号： A61K31/713 ,  A61K48/00 ,  C12N15/85 ,  C12N15/86 ,  C12N2710/16611 ,  C12N2710/16622 ,  C12N2710/16633 ,  C12N2710/16642 ,  C12N2710/16643 ,  C12N2830/007 ,  C12N2830/40 ,  C12N2830/60

摘要： Disclosed are genetic expression cassettes, and vectors comprising them useful for the delivery of isolated nucleic acid segments including those expressing or encoding one or more selected therapeutic constructs (including, without limitation, therapeutic peptides, polypeptides, ribozymes, or catalytic RNA molecules), to one or more selected cells or tissues of a vertebrate animal. Methods employing the disclosed genetic constructs in the development of gene therapy-based viral vector systems are also disclosed. The expression cassettes and viral vectors disclosed herein provide new tools for methods of treating mammalian, and in particular, human diseases, disorders, and/or dysfunctions. The disclosed compositions and methods find particular utility in a variety of investigative, diagnostic, and therapeutic regimens, including, for example, in the treatment or amelioration of symptoms of a variety of mammalian, and particularly, human conditions.

公开(公告)号：US20160120960A1

公开(公告)日：2016-05-05

申请号：US14889750

申请日：2014-05-15

申请人： REGENTS OF THE UNIVERSITY OF MINNESOTA ,  REGENXBIO Inc.

发明人： R. Scott McIvor ,  Lalitha R. Belur ,  Walter Low ,  Carolyn Fairbanks ,  Karen Kozarsky

IPC分类号： A61K38/47 ,  C12N7/00 ,  C12N15/86

CPC分类号： A61K38/47 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/007 ,  C12Y302/01076

摘要： A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.

摘要翻译： 通过鞘内，脑室内或血管内施用编码与该疾病相关的基因产物的rAAV，例如基因产物不存在的哺乳动物来预防，抑制或治疗与中枢神经系统疾病相关的一种或多种症状的方法 或相对于没有疾病的哺乳动物的水平降低。

公开(公告)号：US09023617B2

公开(公告)日：2015-05-05

申请号：US10590136

申请日：2005-02-17

申请人： David C. Bloom ,  Antonio L. Amelio

发明人： David C. Bloom ,  Antonio L. Amelio

IPC分类号： C12N15/86 ,  C12N15/85 ,  A61K48/00

CPC分类号： A61K31/713 ,  A61K48/00 ,  C12N15/85 ,  C12N15/86 ,  C12N2710/16611 ,  C12N2710/16622 ,  C12N2710/16633 ,  C12N2710/16642 ,  C12N2710/16643 ,  C12N2830/007 ,  C12N2830/40 ,  C12N2830/60

摘要： Disclosed are genetic expression cassettes, and vector comprising them useful for the delivery of nucleic acid segments encoding selected therapeutic constructs (including for example, peptides, polypeptides, ribozymes, and catalytic RN molecules), to selected cells and tissues of vertebrate animals. The disclosed genetic constructs are useful in the development of gene therapy vectors, including for example, viral vectors such as HSV, retroviral, lentiviral, AV, and rAAV vectors. The expression cassettes disclosed herein provide new tools in the field of gene therapy, and for the treatment of mammalian, and in particular, human diseases, disorders, and dysfunctions. The disclosed compositions may be utilized in a variety of investigative, diagnostic and therapeutic regimens, including the prevention and treatment of a variety of human diseases.

摘要翻译： 公开了遗传表达盒和包含它们的载体，其可用于将编码所选择的治疗构建体（包括例如肽，多肽，核酶和催化性RN分子）的核酸片段递送至脊椎动物的选定细胞和组织。 所公开的遗传构建体可用于开发基因治疗载体，包括例如病毒载体如HSV，逆转录病毒，慢病毒，AV和rAAV载体。 本文公开的表达盒提供了在基因治疗领域和用于治疗哺乳动物，特别是人类疾病，障碍和功能障碍领域的新工具。 所公开的组合物可以用于多种调查，诊断和治疗方案，包括预防和治疗各种人类疾病。

公开(公告)号：US20150072368A1

公开(公告)日：2015-03-12

申请号：US14375503

申请日：2013-02-08

申请人： Alexandre Fontayne ,  François Coutard

发明人： Alexandre Fontayne ,  François Coutard

IPC分类号： C07K16/28 ,  C12N15/85 ,  C07K16/34

CPC分类号： C07K16/2887 ,  C07K16/34 ,  C12N15/85 ,  C12N2830/007 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/60 ,  C12N2830/85

摘要： Novel transcription units that may be used in expression vectors. The transcription unit allow antibodies to be produced whose gain in productivity is not linked to a particular antigenic target antibody and therefore by extrapolation to a given recombinant protein, nor linked to the culture medium.

摘要翻译： 可用于表达载体的新型转录单位。 转录单元允许产生其生产力增益不与特定抗原靶抗体相关的抗体，因此通过外推到给定的重组蛋白质，也不与培养基连接。

公开(公告)号：US20090214425A1

公开(公告)日：2009-08-27

申请号：US12390096

申请日：2009-02-20

申请人： Daniel Meruelo ,  Jen-Chieh Tseng

发明人： Daniel Meruelo ,  Jen-Chieh Tseng

IPC分类号： A61K51/06 ,  A61K31/7052 ,  A61K49/00 ,  C12Q1/68 ,  C12N15/63 ,  A61P35/00

CPC分类号： C12N7/00 ,  A61K35/768 ,  A61K38/00 ,  A61K38/45 ,  A61K48/0058 ,  A61K51/0491 ,  C12N9/1211 ,  C12N9/78 ,  C12N15/86 ,  C12N2770/36132 ,  C12N2770/36143 ,  C12N2770/36145 ,  C12N2770/36171 ,  C12N2830/007 ,  C12Y207/01021 ,  G01N33/574 ,  G01N2800/52 ,  A61K2300/00

摘要： Disclosed herein are methods for treating a mammal harboring a solid tumor which expresses higher levels of High Affinity Laminin Receptors (LAMR) than normal cells of the same lineage comprising systematically administering to a mammal in need of such treatment a therapeutically effective amount of a Replication Competent (RC) Sindbis virus vector, wherein said vector encodes a suicide gene.

摘要翻译： 本文公开了用于治疗携带实体肿瘤的哺乳动物的方法，所述哺乳动物表达比相同谱系的正常细胞更高水平的高亲和层粘连蛋白受体（LAMR），包括系统地向需要这种治疗的哺乳动物施用治疗有效量的复制主管 （RC）辛德毕斯病毒载体，其中所述载体编码自杀基因。

公开(公告)号：US07011976B1

公开(公告)日：2006-03-14

申请号：US09509591

申请日：1998-03-03

申请人： Andrew S. Little ,  Henry G. Lamparski ,  Daniel R. Henderson ,  Eric R. Schuur

发明人： Andrew S. Little ,  Henry G. Lamparski ,  Daniel R. Henderson ,  Eric R. Schuur

IPC分类号： C12N15/861 ,  C12N15/87 ,  C12N15/33 ,  C12N5/10 ,  C12N5/22 ,  A61K48/00

CPC分类号： C12N15/86 ,  C12N2710/10343 ,  C12N2830/002 ,  C12N2830/007 ,  C12N2830/008

摘要： Replication competent adenoviral vectors specific for cells expressing alfa-fetoprotein (AFP) are provided. These replication-competetent adenoviral vectors comprise adenovirus genes essential for replication under the transcriptional control of an AFP-transcriptional regulatory element.

摘要翻译： 提供了对表达甲胎蛋白（AFP）的细胞特异性的复制感受态腺病毒载体。 这些复制竞争性腺病毒载体包含在AFP转录调控元件的转录控制下复制必需的腺病毒基因。

公开(公告)号：US20030095989A1

公开(公告)日：2003-05-22

申请号：US10023969

申请日：2001-12-17

发明人： John M. Irving ,  Jane S. Lebkowski

IPC分类号： A61K048/00 ,  C12Q001/70 ,  C12N007/01 ,  C12N005/08 ,  A01N063/00 ,  A61K039/12 ,  A61K039/245 ,  A61K039/25 ,  A61K039/23 ,  A61K039/235 ,  C12N007/00 ,  C12N015/00 ,  C12N015/09 ,  C12N015/63 ,  C12N015/70 ,  C12N015/74 ,  C12N005/00 ,  C12N005/02

CPC分类号： C12N7/00 ,  A61K35/761 ,  C07K14/005 ,  C12N15/86 ,  C12N2710/10332 ,  C12N2710/10343 ,  C12N2710/10345 ,  C12N2710/16122 ,  C12N2710/20022 ,  C12N2830/007 ,  C12N2830/008

摘要： Described in this disclosure is a system for gene therapy using a chimeric vector made from an adenovirus genome and a heterologous gene that functionally replaces an adenovirus gene required for replication or assembly. Cytolytic viruses can be produced that target particular tissue types by virtue of having replication controlled by a specific transcription control elementnullsuch as the promoter for telomerase reverse transcriptase. These therapeutic viruses are believed to have an improved safety and efficacy profile compared with previously available systems.

摘要翻译： 在本公开中描述的是使用由腺病毒基因组和异源基因构成的嵌合载体进行基因治疗的系统，其功能性替代复制或组装所需的腺病毒基因。 可以通过由特异性转录控制元件（例如端粒酶逆转录酶的启动子）控制复制而产生靶向特定组织类型的细胞溶解病毒。 与以前可用的系统相比，这些治疗性病毒被认为具有改进的安全性和功效特征。